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Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase .
Sio Gene TherapiesDecember 15, 2020 GMT
-  Generally well-tolerated with a favorable safety profile in five patients
-  Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer
-  At Month 6, enzyme activity restored to 23-57% (mean: 38%) of normal reference levels
-  All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression (CGI) scales ....

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